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A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation

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ClinicalTrials.gov Identifier: NCT02725567
Recruitment Status : Recruiting
First Posted : April 1, 2016
Last Update Posted : January 16, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE March 14, 2016
First Posted Date  ICMJE April 1, 2016
Last Update Posted Date January 16, 2019
Actual Study Start Date  ICMJE March 2016
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 31, 2016)
  • Part A: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [ Time Frame: Day 1 up to Day 70 ]
  • Part B: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [ Time Frame: Day 1 up to Week 24 ]
  • Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
  • Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02725567 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 31, 2016)
  • Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
  • Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
  • Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [ Time Frame: through Week 24 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation
Official Title  ICMJE A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation
Brief Summary The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: ivacaftor
Other Name: Kalydeco
Study Arms  ICMJE
  • Experimental: Part A
    • Group 1: Participants 12 to < 24 months
    • Group 2: Participants 6 to < 12 months (enrollment begins after an assessment of data from Group 1)
    • Group 3: Participants 3 to < 6 months (enrollment begins after an assessment of data from Group 2)
    • Group 4: Participants 0 to < 3 months (enrollment begins after an assessment of data from Group 3)
    Intervention: Drug: ivacaftor
  • Experimental: Part B
    • Group 5: Participants 12 to < 24 months (enrollment begins after an assessment of data from Part A, Group 1
    • Group 6: Participants 6 to < 12 months (enrollment begins after an assessment of data from Part A, Group 2
    • Group 7: Participants 0 to < 6 months (enrollment begins after an assessment of data from Part A, Group 3, and also enrollment for subjects < 3 months begins after review of data from Part A Group 4)
    Intervention: Drug: ivacaftor
Publications * Rosenfeld M, Wainwright CE, Higgins M, Wang LT, McKee C, Campbell D, Tian S, Schneider J, Cunningham S, Davies JC; ARRIVAL study group. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018 Jul;6(7):545-553. doi: 10.1016/S2213-2600(18)30202-9. Epub 2018 Jun 7. Erratum in: Lancet Respir Med. 2018 Jul;6(7):e35. Erratum in: Lancet Respir Med. 2019 Apr;7(4):e15.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 6, 2016)
35
Original Estimated Enrollment  ICMJE
 (submitted: March 31, 2016)
30
Estimated Study Completion Date  ICMJE June 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Confirmed diagnosis of CF by sweat chloride value or CF mutation criteria.
  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
  • Hematology, serum chemistry, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator.

Exclusion Criteria:

  • History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
  • Colonization with organisms associated with a more rapid decline in pulmonary status at screening
  • History of abnormal liver function or abnormal liver function at screening
  • History of solid organ or hematological transplantation
  • Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
  • Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening
  • Hemoglobin (Hgb) <9.5 g/dL at screening
  • Chronic kidney disease of Stage 3 or above
  • Presence of a non-congenital or progressive lens opacity or cataract at Screening

Other protocol defined Inclusion/Exclusion Criteria may apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 24 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com
Listed Location Countries  ICMJE Australia,   Canada,   Ireland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02725567
Other Study ID Numbers  ICMJE VX15-770-124
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP